MX2019006157A - Prevención de la distrofia muscular mediante la edición de genes mediada por crispr/cpf1. - Google Patents
Prevención de la distrofia muscular mediante la edición de genes mediada por crispr/cpf1.Info
- Publication number
- MX2019006157A MX2019006157A MX2019006157A MX2019006157A MX2019006157A MX 2019006157 A MX2019006157 A MX 2019006157A MX 2019006157 A MX2019006157 A MX 2019006157A MX 2019006157 A MX2019006157 A MX 2019006157A MX 2019006157 A MX2019006157 A MX 2019006157A
- Authority
- MX
- Mexico
- Prior art keywords
- editing
- myo
- dystrophin
- mediated
- mutations
- Prior art date
Links
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/102—Mutagenizing nucleic acids
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4707—Muscular dystrophy
- C07K14/4708—Duchenne dystrophy
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0603—Embryonic cells ; Embryoid bodies
- C12N5/0606—Pluripotent embryonic cells, e.g. embryonic stem cells [ES]
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0696—Artificially induced pluripotent stem cells, e.g. iPS
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases RNAses, DNAses
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0306—Animal model for genetic diseases
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2800/00—Nucleic acids vectors
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Biophysics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Neurology (AREA)
- Developmental Biology & Embryology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Cell Biology (AREA)
- Reproductive Health (AREA)
- Gynecology & Obstetrics (AREA)
- Virology (AREA)
- Epidemiology (AREA)
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- Transplantation (AREA)
- Dermatology (AREA)
- Crystallography & Structural Chemistry (AREA)
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201662426853P | 2016-11-28 | 2016-11-28 | |
PCT/US2017/063468 WO2018098480A1 (fr) | 2016-11-28 | 2017-11-28 | Prévention de la dystrophie musculaire par édition de gène médiée par crispr/cpf1 |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2019006157A true MX2019006157A (es) | 2019-11-21 |
Family
ID=60703123
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2019006157A MX2019006157A (es) | 2016-11-28 | 2017-11-28 | Prevención de la distrofia muscular mediante la edición de genes mediada por crispr/cpf1. |
Country Status (8)
Country | Link |
---|---|
US (1) | US20200046854A1 (fr) |
EP (1) | EP3545090A1 (fr) |
JP (1) | JP2019536782A (fr) |
CN (1) | CN110382695A (fr) |
AU (1) | AU2017364106A1 (fr) |
CA (1) | CA3044531A1 (fr) |
MX (1) | MX2019006157A (fr) |
WO (1) | WO2018098480A1 (fr) |
Families Citing this family (45)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2013066438A2 (fr) | 2011-07-22 | 2013-05-10 | President And Fellows Of Harvard College | Évaluation et amélioration de la spécificité de clivage des nucléases |
EP2841572B1 (fr) | 2012-04-27 | 2019-06-19 | Duke University | Correction génétique de gènes ayant subi une mutation |
US9163284B2 (en) | 2013-08-09 | 2015-10-20 | President And Fellows Of Harvard College | Methods for identifying a target site of a Cas9 nuclease |
US9359599B2 (en) | 2013-08-22 | 2016-06-07 | President And Fellows Of Harvard College | Engineered transcription activator-like effector (TALE) domains and uses thereof |
US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
US20150165054A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
US10077453B2 (en) | 2014-07-30 | 2018-09-18 | President And Fellows Of Harvard College | CAS9 proteins including ligand-dependent inteins |
US11970710B2 (en) | 2015-10-13 | 2024-04-30 | Duke University | Genome engineering with Type I CRISPR systems in eukaryotic cells |
EP3365357B1 (fr) | 2015-10-23 | 2024-02-14 | President and Fellows of Harvard College | Protéines cas9 évoluées pour l'édition génétique |
EP3487523B1 (fr) * | 2016-07-19 | 2023-09-06 | Duke University | Applications thérapeutiques de l'édition du génome fondée sur cpf1 |
AU2017306676B2 (en) | 2016-08-03 | 2024-02-22 | President And Fellows Of Harvard College | Adenosine nucleobase editors and uses thereof |
AU2017308889B2 (en) | 2016-08-09 | 2023-11-09 | President And Fellows Of Harvard College | Programmable Cas9-recombinase fusion proteins and uses thereof |
WO2020225754A1 (fr) * | 2019-05-06 | 2020-11-12 | Mcmullen Tara | Édition de gènes crispr pour maladies autosomiques dominantes |
US11542509B2 (en) | 2016-08-24 | 2023-01-03 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
SG11201903089RA (en) | 2016-10-14 | 2019-05-30 | Harvard College | Aav delivery of nucleobase editors |
WO2018119359A1 (fr) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Édition du gène récepteur ccr5 pour protéger contre l'infection par le vih |
WO2018165504A1 (fr) | 2017-03-09 | 2018-09-13 | President And Fellows Of Harvard College | Suppression de la douleur par édition de gène |
US11542496B2 (en) | 2017-03-10 | 2023-01-03 | President And Fellows Of Harvard College | Cytosine to guanine base editor |
IL306092A (en) | 2017-03-23 | 2023-11-01 | Harvard College | Nucleic base editors that include nucleic acid programmable DNA binding proteins |
WO2018179578A1 (fr) * | 2017-03-30 | 2018-10-04 | 国立大学法人京都大学 | Procédé pour induire un saut d'exon par édition génomique |
WO2018209320A1 (fr) | 2017-05-12 | 2018-11-15 | President And Fellows Of Harvard College | Arn guides incorporés par aptazyme pour une utilisation avec crispr-cas9 dans l'édition du génome et l'activation transcriptionnelle |
JP2020534795A (ja) | 2017-07-28 | 2020-12-03 | プレジデント アンド フェローズ オブ ハーバード カレッジ | ファージによって支援される連続的進化(pace)を用いて塩基編集因子を進化させるための方法および組成物 |
WO2019139645A2 (fr) | 2017-08-30 | 2019-07-18 | President And Fellows Of Harvard College | Éditeurs de bases à haut rendement comprenant une gam |
US11795443B2 (en) | 2017-10-16 | 2023-10-24 | The Broad Institute, Inc. | Uses of adenosine base editors |
US20210308273A1 (en) | 2018-08-02 | 2021-10-07 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
KR20210081324A (ko) | 2018-08-02 | 2021-07-01 | 다인 세라퓨틱스, 인크. | 근육 표적화 복합체 및 안면견갑상완 근육 이영양증을 치료하기 위한 그의 용도 |
US20220017888A1 (en) | 2018-12-11 | 2022-01-20 | Kyoto University | Method for inducing deletion in genomic dna |
EA202192233A1 (ru) * | 2019-02-12 | 2021-11-11 | Университа' Дельи Студи Ди Тренто | МОЛЕКУЛЫ ГИДОВОЙ РНК Cas12a И ИХ ПРИМЕНЕНИЕ |
JP2022526908A (ja) | 2019-03-19 | 2022-05-27 | ザ ブロード インスティテュート,インコーポレーテッド | 編集ヌクレオチド配列を編集するための方法および組成物 |
CN110499333A (zh) * | 2019-08-01 | 2019-11-26 | 广州德赫生物科技有限公司 | 用于修复dmd基因突变的核酸序列及系统 |
WO2021216674A1 (fr) * | 2020-04-24 | 2021-10-28 | University Of Massachusetts | Plateformes d'édition génétique thérapeutique à médiation par cas 12a/nls améliorées |
AU2021267940A1 (en) | 2020-05-08 | 2022-12-08 | President And Fellows Of Harvard College | Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence |
CA3178591A1 (fr) * | 2020-05-12 | 2021-11-18 | Courtney Young | Agents immunosuppresseurs et methodes de re-dosage d'administration virale pour therapie genique |
US20220096606A1 (en) * | 2020-09-09 | 2022-03-31 | Vertex Pharmaceuticals Incorporated | Compositions and Methods for Treatment of Duchenne Muscular Dystrophy |
WO2022140340A1 (fr) * | 2020-12-22 | 2022-06-30 | Vertex Pharmaceuticals Incorporated | Compositions comprenant un guide d'arn ciblant dmd et leurs utilisations |
WO2022251181A1 (fr) * | 2021-05-25 | 2022-12-01 | The Board Of Regents Of The University Of Texas System | Correction des mutations de la dystrophie musculaire de duchenne à l'aide de crispr à une seule coupure délivrée par un virus adéno-associé |
US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
CA3226307A1 (fr) * | 2021-07-09 | 2023-01-12 | Dyne Therapeutics, Inc. | Complexes de ciblage musculaire et leurs utilisations pour le traitement de dystrophinopathies |
US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
CN114214360A (zh) * | 2021-12-27 | 2022-03-22 | 西安英创生物技术有限公司 | 先天性肌无力小鼠模型、其构建方法及应用 |
EP4215614A1 (fr) | 2022-01-24 | 2023-07-26 | Dynacure | Thérapie de combinaison pour maladies liées à la dystrophine |
WO2023172927A1 (fr) * | 2022-03-08 | 2023-09-14 | Vertex Pharmaceuticals Incorporated | Excisions précises de parties d'exon 44, 50 et 53 pour le traitement de la dystrophie musculaire de duchenne |
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Publication number | Priority date | Publication date | Assignee | Title |
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DK99653C (da) | 1959-06-24 | 1964-08-31 | Skaanska Aettikfabriken Ab | Pynteliste til brug som sammenfojningsliste, hjørneliste eller endeliste ved montering af beklædningsplader. |
EP0273085A1 (fr) | 1986-12-29 | 1988-07-06 | IntraCel Corporation | Procédé pour faire entrer des acides nucléiques dans des cellules eucaryotes |
EP2350281B1 (fr) * | 2008-10-24 | 2014-05-14 | Sarepta Therapeutics, Inc. | Compositions à sauts d'exons multiples pour la dmd |
LT3401400T (lt) | 2012-05-25 | 2019-06-10 | The Regents Of The University Of California | Būdai ir kompozicijos, skirtos rnr molekulės nukreipiamai tikslinės dnr modifikacijai ir rnr molekulės nukreipiamam transkripcijos moduliavimui |
CA2906812A1 (fr) * | 2013-03-15 | 2014-09-18 | Sarepta Therapeutics, Inc. | Compositions ameliorees pour le traitement de la dystrophie musculaire |
EP3417880A1 (fr) * | 2013-06-05 | 2018-12-26 | Duke University | Édition et régulation géniques à guidage arn |
ES2821149T3 (es) * | 2014-03-12 | 2021-04-23 | Prec Biosciences Inc | Eliminación del exón del gen de la distrofina mediante nucleasas modificadas genéticamente |
CA2959130A1 (fr) * | 2014-08-11 | 2016-02-18 | The Board Of Regents Of The University Of Texas System | Prevention de la dystrophie musculaire par edition de gene mediee par crispr/cas9 |
WO2016025468A2 (fr) * | 2014-08-11 | 2016-02-18 | The Board Of Trustees Of The University Of Illinois | Dispositifs et procédés associés pour la caractérisation épidermique de fluides biologiques |
US9790490B2 (en) * | 2015-06-18 | 2017-10-17 | The Broad Institute Inc. | CRISPR enzymes and systems |
ES2929110T3 (es) * | 2015-08-25 | 2022-11-24 | Univ Duke | Composiciones y métodos para mejorar la especificidad en ingeniería genética usando endonucleasas guiadas por ARN |
WO2017049407A1 (fr) * | 2015-09-23 | 2017-03-30 | UNIVERSITé LAVAL | Modification du gène de la dystrophine et ses utilisations |
US11369692B2 (en) * | 2015-10-28 | 2022-06-28 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of Duchenne Muscular Dystrophy |
EP3487523B1 (fr) * | 2016-07-19 | 2023-09-06 | Duke University | Applications thérapeutiques de l'édition du génome fondée sur cpf1 |
-
2017
- 2017-11-28 US US16/464,124 patent/US20200046854A1/en not_active Abandoned
- 2017-11-28 EP EP17817498.3A patent/EP3545090A1/fr not_active Withdrawn
- 2017-11-28 CA CA3044531A patent/CA3044531A1/fr active Pending
- 2017-11-28 JP JP2019528478A patent/JP2019536782A/ja active Pending
- 2017-11-28 WO PCT/US2017/063468 patent/WO2018098480A1/fr unknown
- 2017-11-28 MX MX2019006157A patent/MX2019006157A/es unknown
- 2017-11-28 AU AU2017364106A patent/AU2017364106A1/en active Pending
- 2017-11-28 CN CN201780084906.8A patent/CN110382695A/zh active Pending
Also Published As
Publication number | Publication date |
---|---|
WO2018098480A1 (fr) | 2018-05-31 |
CA3044531A1 (fr) | 2018-05-31 |
JP2019536782A (ja) | 2019-12-19 |
AU2017364106A1 (en) | 2019-06-20 |
CN110382695A (zh) | 2019-10-25 |
US20200046854A1 (en) | 2020-02-13 |
EP3545090A1 (fr) | 2019-10-02 |
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